Deliberative processes in health technology assessment of medicines: the case of Spain.

OBJECTIVES: Spain incorporated in 2020 changes in its health technology assessment (HTA), pricing, and reimbursement system for medicines including publishing reports, development of networks of experts, or consultation with stakeholders. Despite these changes, it is unclear how deliberative frameworks are applied and the process has been criticized for not being sufficiently transparent. This study analyses the level of implementation of deliberative processes in HTA for medicines in Spain. METHODS: We review the grey literature and summarize the Spanish HTA, pricing, and reimbursement process of medicines. We apply the deliberative processes for HTA checklist, developed to assess the overall context of the deliberative process, and identify the stakeholders involved and type of involvement following the framework for evidence-informed deliberative processes, a framework for benefit package design that aims to optimize the legitimacy of decision making. RESULTS: In the Spanish HTA, pricing, and reimbursement process deliberation takes place in order to exchange viewpoints and reach common ground, mainly during the prioritization, assessment, and appraisal steps. It is closed to the public, not clearly summarized in published documents and limited to the Ministry of Health, the regulatory agency, other Ministries, and experts with mostly clinical and/or pharmaceutical background. The views of stakeholders are only represented through consultation. Communication is the most commonly used form of stakeholder engagement. CONCLUSIONS: Despite improvements in transparency of the Spanish HTA process for evaluating medicines, aspects related to stakeholder involvement and implementation of deliberative frameworks need further attention in order to achieve further legitimacy of the process.

Institutionalizing health technology assessment in Egypt: Situational analysis and roadmap.

Objective: To conduct a situational analysis with the aim to inform future health technology assessment efforts (HTA) in Egypt. Introduction: The Egyptian government has set universal health coverage as a 2030 target. Several agencies have been created in the context of the ongoing healthcare reform. The Egyptian Authority for Unified Procurement, Medical Supply and the Management of Medical Technology (UPA) is one of them and was established to support strategic procurement using HTA. Methods: Description of the development of HTA in Egypt supported by a literature search as part of a scoping exercise, and a stakeholder analysis and identification of HTA capacity survey, based on previous surveys, with relevant stakeholders conducted in 2022. This was followed by a stakeholder event where results were shared and further contextualized. Results: The UPA is expected to evaluate the cost-effectiveness of health technologies and public health programs. The HTA process is being developed, focusing on the assessment of the value of new pharmaceuticals being introduced to the Egyptian market. A total of 16 participants responded on behalf of their organizations to the stakeholder analysis and identification of HTA capacity survey. More than 80% of the respondents were familiar with current efforts conducted by UPA and strongly support the implementation of HTA in Egypt. Transparency was highlighted as an important criterion. Over 90% of the respondents mentioned economic analyses as an HTA product being developed in Egypt, and medicines were the type of technology that stakeholders ranked as first in the rank of health technologies that need the output from HTA urgently. Capability building and training were highlighted as areas in which further support is required. Conclusion: This study represents the first attempt to describe the current path for HTA in Egypt. There seems to be momentum in Egypt to proceed and advance with HTA institutionalization. It would be important that next steps are built on the skills and capabilities already in place in Egypt, ensure methods and processes are in place and up to date and involve the wider system in Egypt so stakeholders can appropriately contribute and participate in the HTA process.

NICE's evaluations of medicines authorized by EMA with conditional marketing authorization or under exceptional circumstances.

OBJECTIVES: To investigate the impact of the uncertainty stemming from products with European conditional marketing authorization (CMA) or authorization in exceptional circumstances (AEC) on the National Institute for Health and Care Excellence's (NICE) recommendations. METHODS: Products which received CMA/AEC by European Medicines Agency (EMA) up to 1 December 2016 were identified and matched with corresponding NICE decisions issued by August 2017, the status of which was then traced to August 2019. We assessed whether the conversion of CMA to full marketing authorization triggered a review of a NICE decision. The odds of a recommendation carrying a commercial arrangement for products with and without CMA/AEC were calculated. RESULTS: Fifty-four products were granted CMA/AEC by EMA. NICE conducted thirty evaluations of products with CMA/AEC. Twelve products were recommended by NICE by August 2017 and fourteen by August 2019. All recommendations had an associated commercial arrangement. The odds of carrying a commercial arrangement were higher for products with CMA/AEC compared to those with full authorization. Conversions from conditional to full authorization among products not recommended by NICE did not trigger an appraisal review. CONCLUSIONS: Uncertainty, stemming from the lack of robust clinical data of products authorized with CMA/AEC, has a substantial impact on HTA recommendations, frequently requiring risk mitigation mechanisms such as commercial and data collection arrangements. Further analyses should be conducted to assess whether the benefits of early access strategies outweigh the risks for patients and the healthcare system.

Integrating health technology assessment requirements in the clinical development of medicines: the experience from NICE scientific advice.

PURPOSE: The primary objective of the study was to analyse the proposed clinical development and economic evaluation plans for investigational medicinal products for which pharmaceutical companies have sought health technology assessment (HTA) scientific advice (SA). METHODS: We have selected and analysed all the scientific advice procedures undertaken by National Institute for Health and Care Excellence (NICE) SA between 1 January 2009 and 3 December 2015 for investigational medicinal products. We have mapped the questions asked by the companies and the areas of advice highlighted in the advice reports to the sections of the NICE methods guide to the technology appraisals (2013). RESULTS: An overwhelming proportion of SA procedures have addressed questions related to the clinical development and specifically the main pivotal efficacy studies. Approximately a quarter of the questions relate to the approaches to economic evaluation. Questions raised in European Medicines Agency-HTA procedures generally focus on clinical efficacy issues whereas cost-effectiveness ones tend to dominate in NICE-only procedures. Our analysis shows that the issues mostly discussed in the HTA SA are the choice of comparator, the generalisability of the clinical trial evidence to the NHS practice and the impact of the clinical trial outcomes on quality of life and survival. Less disagreement with the developers' plans was seen in the choice of clinical endpoints, population definition, position of the technology in the treatment pathway and study design. CONCLUSIONS: Scientific advice is designed to improve the quality of evidence and approaches to evidence generation for future regulatory approval and HTA evaluation. Our experience to date suggests that payer requirements are inconsistently integrated in the clinical development programmes. More efforts should be dedicated to demonstrating the clinical value of new medicinal products to patients and key decision-makers.

Economic evaluations of implantable cardioverter defibrillators: a systematic review.

The aim of this paper was to review the cost-effectiveness studies of implantable cardioverter defibrillators (ICD) for primary or secondary prevention of sudden cardiac death (SCD). A systematic review of the literature published in English or Spanish was performed by electronically searching MEDLINE and MEDLINE in process, EMBASE, NHS-EED, and EconLit. Some keywords were implantable cardioverter defibrillator, heart failure, heart arrest, myocardial infarction, arrhythmias, syncope, sudden death. Selection criteria were the following: (1) full economic evaluations published after 1995, model-based studies or alongside clinical trials (2) that explored the cost-effectiveness of ICD with or without associated treatment compared with placebo or best medical treatment, (3) in adult patients for primary or secondary prevention of SCD because of ventricular arrhythmias. Studies that fulfilled these criteria were reviewed and data were extracted by two reviewers. The methodological quality of the studies was assessed and a narrative synthesis was prepared. In total, 24 studies were included: seven studies on secondary prevention and 18 studies on primary prevention. Seven studies were performed in Europe. For secondary prevention, the results showed that the ICD is considered cost-effective in patients with more risk. For primary prevention, the cost-effectiveness of ICD has been widely studied, but uncertainty about its cost-effectiveness remains. The cost-effectiveness ratios vary between studies depending on the patient characteristics, methodology, perspective, and national settings. Among the European studies, the conclusions are varied, where the ICD is considered cost-effective or not dependent on the study.

Revisión sistemática de utilidades relacionadas con la salud en España: el caso de la salud mental / Systematic review of health-related utilities in Spain: the case of mental health

Objetivo Dada la carencia de un registro de utilidades en población española para su uso en evaluaciones económicas, se desarrolló una revisión sistemática de utilidades o preferencias por estados de salud obtenidas de población española con el fin último de crear un catálogo accesible a los investigadores. En este artículo se detallan las utilidades relacionadas con la salud mental. Método Revisión sistemática de la literatura, con búsquedas en MEDLINE, CRD, Embase, PsycINFO, CINAHL, Cochrane y otras bases de datos. La estrategia de búsqueda combinó términos relacionados con utilidades y con España. Los criterios de inclusión eran población residente, afectada o no por alguna enfermedad; utilidades valoradas con cuestionarios validados en España (EQ-5D, HUI, SF-6D) o siguiendo técnicas directas (equivalencia temporal, standard gamble, escala visual analógica). Se realizó una síntesis narrativa de los artículos incluidos, detallando los resultados relacionados con problemas de salud mental. Resultados Se incluyeron 103 estudios, de los que se extrajeron 742 utilidades. Se identificaron 69 utilidades relacionadas con trastornos mentales y del comportamiento, extraídas de 12 estudios. El instrumento más empleado fue el cuestionario EQ-5D. Gran parte de los artículos excluidos evaluaba la calidad de vida, pero sin ofrecer el valor de la utilidad. Conclusiones La presente revisión añade valor al estudio de las utilidades en España mediante la recopilación de valores que puedan ser incluidos en evaluaciones económicas, permitiendo además identificar lagunas en la investigación en este campo. Los valores relacionados con la salud mental identificados se asemejan a otros informados en la literatura internacional (AU)

Objectives Currently, there is no registry of utility values for the Spanish population that could potentially be used in economic evaluations. Consequently, a systematic review of utilities or preferences for health states in the Spanish population was conducted. The results related to mental health are reported. Methods A systematic review of the literature was conducted. The main databases searched were MEDLINE, CRD, Embase, PsycINFO, CINAHL, and Cochrane. The search strategy combined terms related to utilities and Spain. The inclusion criteria comprised the resident population in Spain, whether affected by any disease or not; the reported utilities had to be evaluated through a tool validated in Spain (i.e., EQ-5D, HUI, SF-6D) and/or following accepted techniques (e.g., time trade-off, standard gamble, or the visual analog scale). A narrative synthesis of articles was undertaken and the results related to mental health summarized. Results A total of 103 articles were finally included, from which 742 utility values were extracted. Sixty-nine utility values related to mental health and behavioral disorders were extracted from 12 studies. The most widely used tool was the E5-QD questionnaire. Most of the excluded articles evaluated quality of life but did not provide an estimation of utilities. Conclusions This review adds value to research on utilities in Spain by gathering values to be included in economic evaluations, as well as by identifying research gaps in this field. The utility values related to mental health identified in this study are similar to those reported in international publications (AU)

[Systematic review of health-related utilities in Spain: the case of mental health]. / Revisión sistemática de utilidades relacionadas con la salud en España: el caso de la salud mental.

OBJECTIVES: Currently, there is no registry of utility values for the Spanish population that could potentially be used in economic evaluations. Consequently, a systematic review of utilities or preferences for health states in the Spanish population was conducted. The results related to mental health are reported. METHODS: A systematic review of the literature was conducted. The main databases searched were MEDLINE, CRD, Embase, PsycINFO, CINAHL, and Cochrane. The search strategy combined terms related to utilities and Spain. The inclusion criteria comprised the resident population in Spain, whether affected by any disease or not; the reported utilities had to be evaluated through a tool validated in Spain (i.e., EQ-5D, HUI, SF-6D) and/or following accepted techniques (e.g., time trade-off, standard gamble, or the visual analog scale). A narrative synthesis of articles was undertaken and the results related to mental health summarized. RESULTS: A total of 103 articles were finally included, from which 742 utility values were extracted. Sixty-nine utility values related to mental health and behavioral disorders were extracted from 12 studies. The most widely used tool was the E5-QD questionnaire. Most of the excluded articles evaluated quality of life but did not provide an estimation of utilities. CONCLUSIONS: This review adds value to research on utilities in Spain by gathering values to be included in economic evaluations, as well as by identifying research gaps in this field. The utility values related to mental health identified in this study are similar to those reported in international publications.